Hundreds of children with ultra-aggressive cancers have had their tumours shrunk or eradicated completely in a world-first program giving hope to every family with a child diagnosed with cancer in Australia.
The Children’s Cancer Institute and the Kids Cancer Centre at Sydney Children’s Hospital launched the zero childhood cancer program in 2017 to provide personalised treatments to children whose cancer had relapsed or did not respond to standard therapies.
Sean and Edwina Bradstreet say their daughter Scout is unrecognisable from the girl born with a brain tumour less than two years ago. Credit: Janie Barrett
Now, the program is open to every child diagnosed with cancer in Australia, and the first study analysing its success published in the international journal Nature Medicine on Thursday.
Of 256 children given a targeted treatment based on a molecular analysis of their cancer, 55 per cent had their tumour either disappear completely or shrink by at least half over six months. After two years, 26 per cent of children who received personalised therapies survived, more than double the rate of children on the standard treatment.
“For these children, there generally is no standard therapy available to them, so to see any response in this setting is very exciting,” said Professor David Ziegler, who oversees the program’s clinical trials and was one of the lead authors on the study. “More children are alive and have their disease under control using this approach. That’s really groundbreaking, and something no one’s really been able to show before.”
One of Ziegler’s patients, Scout Bradstreet, was just one month old when doctors found a tumour on the right side of her brain.
Surgeons removed the tumour and sent it off for genomic analysis, while Scout started on chemotherapy to fight the high-grade glioma. It took a massive toll on her young body.
“She was always so sick, we would be home for three days if we were lucky, and then we’d be back in hospital for two weeks,” her mother Edwina said.
The genomic analysis found Scout’s cancer was linked to a gene mutation known as an ALK fusion, allowing scientists to test in the lab how it would react to an ALK-inhibiting drug called alectinib.
Promising results provided a lifeline for the Bradstreets after seven months of chemotherapy failed to stop Scout’s tumour from growing. When she began the treatment in November, her reaction was almost immediate.
Recent scans show the tumour has disappeared. A medicine dissolved into water and given to her in apple juice twice a day helps to keep the cancer from returning – a small sacrifice compared to endless hospital visits for intense chemotherapy.
“It’s a different child who’s with us now,” father Sean said. “If you looked at her today, you wouldn’t know there was anything going on.”
Melbourne father Mohammed Haddara was initially reluctant to subject his family to genetic testing after doctors found a tumour in his daughter Daad’s brain.
But after the nine-year-old’s tumour returned a week and a half after she had it surgically removed, and her older sister Mounawar also fell sick, genetic testing confirmed he and wife Rima were both carriers for a gene predisposing their children to a spectrum of childhood malignancies.
The discovery helped her doctor find an immunotherapy treatment which has destroyed the tumour in Daad’s brain and helped her return to life as normal.
“She’s energetic [again], she likes to ride her bike, she likes to go to school … she loves sports,” Mohammed Haddara said. “Nothing’s a guarantee in life, but when they gave it [the immunotherapy] to her, bang – it worked perfectly.”
Rima Haddara with 10-year-old daughter Daad, whose brain tumour has shrunk to almost nothing thanks to a personalised immunotherapy. Credit: Joe Armao
All nine of Australia’s children’s hospitals are involved in the program, and all genetic testing is free.
Medicines are funded by a mix of charities, clinical trials, and philanthropic support from pharmaceutical companies and hospitals.
Ziegler, from the Children’s Cancer Institute, said the program would continue to track children to see how they responded to the therapies long-term.
“They say saving one life is like saving a universe,” he said. “That is literally how it feels to see even one child who is doing well, where previously they would have had no hope.”
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